Effects on FDA Morale and the Potential for en masse Resignations of that Agency’s Reviewers

In a previous blog we identified several critical processes that could be affected if there were major changes in the laws underpinning FDA review and approval of new drugs, biological therapies and vaccines.

Should Congress make major modifications in the Food, Drug and Cosmetics and Public Health Service Acts, the requirement for substantial evidence of both efficacy and safety could be altered. The resulting shift in assessment of risk/benefit could have a profound effect on the organization, procedures, internal guidance, workload, and approach of Agency Reviewers.

Over the last several years these Reviewers have increasingly been hired by the FDA from industry, university research, and the practice of medicine, thus incorporating their experience outside of the government environment and enriching the Agency with sophisticated, experienced, realistic and sound resources. Along with the traditional facilitated approaches to accelerating the medical product review process (e.g., Fast Track, Accelerated Approval, and Orphan Drug Designation), newer programs such as Breakthrough Designation and Regenerative Medicine Advanced Therapy Designation have responded to critical healthcare emergencies and added to the officially sanctioned means for bringing new therapeutic interventions to patients with unmet medical need, while still meeting well-defined standards for safety and efficacy.

We believe that a radical change in the requirements for substantial evidence of both safety and efficacy, if introduced into the FDA, will lead to a massive upheaval in both the morale of Agency Reviewers, and concerns for the overall health of the system for development, review and approval of new diagnostic and therapeutic interventions.

This could also have a significant adverse effect both on the US system for reimbursement and worldwide due to the fallout over the lowered competitiveness of products approved or licensed under inferior standards of risk/benefit. The FDA has often been thought of as a “gold standard” for review of new chemical and biological entities, emulated by regulatory authorities worldwide. Should the US system of substantial evidence of both efficacy and safety imposed prior to marketing approval of new products be compromised, ex-US regulators and reimbursement authorities could well place such products on a second or third tier for accessibility, thus adversely affecting US firms who have developed them under the new legal/regulatory scheme.